NCT03128593已完成1 期
A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 已完成
- 入组人数
- 14
- 试验地点
- 8
- 主要终点
- Number of participants with Adverse Events
研究概览
简要总结
The purpose of this study in patients with mucopolysaccharidosis type II (MPS II) is below,
- to collect the safety information of JR-141
- to evaluate the plasma pharmacokinetics of JR-141
- to explore the efficacy of JR-141 on MPS II-related central nervous system symptoms and general symptoms
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Patients aged 6 years or older at the time of informed consent.
- •Patients diagnosed with MPS II.
- •Patients who have received idursulfase (0.5 mg/kg/week) continuously for at least 12 weeks until the initial dose of JR-141.
排除标准
- •Patients with a history of hematopoietic stem cell transplantation, excluding those who need enzyme replacement therapy even after hematopoietic stem cell transplantation.
- •Patients in whom lumbar puncture cannot be performed.
- •Patients who have developed serious drug allergy or hypersensitivity that is inappropriate for participation in the study.
- •Patients who have received other investigational products within 4 months before enrollment in the study.
研究组 & 干预措施
Experimental: JR-141
Experimental
干预措施: JR-141 (Drug)
结局指标
主要结局
Number of participants with Adverse Events
时间窗: 4 weeks
* Adverse events * Laboratory tests * Vital signs * 12-lead electrocardiogram * Antibody * Infusion associated reaction
次要结局
- Urinary and serum heparan sulfate (HS) /dermatan sulfate (DS)(4 weeks)
- HS/DS in CSF(4 weeks)
- Plasma Pharmacokinetic parameter [Maximum Plasma Concentration [Cmax]](4 weeks)
- Urinary total GAG(4 weeks)
- Plasma Pharmacokinetic parameter [Area Under the Curve [AUC]](4 weeks)
- Liver and spleen volumes(4 weeks)
- Cardiac function(4 weeks)
研究者
研究点 (8)
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